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dc.contributor.authorViscomi, CF
dc.contributor.authorGarone, Caterina
dc.date.accessioned2018-12-01T00:31:23Z
dc.date.available2018-12-01T00:31:23Z
dc.date.issued2018-10-19
dc.identifier.issn1470-8752
dc.identifier.urihttps://www.repository.cam.ac.uk/handle/1810/286221
dc.description.abstractPreclinical work aimed at developing new therapies for mitochondrial diseases has recently given new hopes and opened unexpected perspectives for the patients affected by these pathologies. In contrast, only minor progresses have been achieved so far in the translation into the clinics. Many challenges are still ahead, including the need for a better characterization of the pharmacological effects of the different approaches and the design of appropriate clinical trials with robust outcome measures for this extremely heterogeneous, rare, and complex group of disorders. In this review, we will discuss the most important achievements and the major challenges in this very dynamic research field.
dc.description.sponsorshipOur work is supported by MRC (grants: MC_UU_00015/5 and MC_EX_MR/P007031/1) and ERC (grant FP7-322424)
dc.publisherPortland Press
dc.rightsAttribution 4.0 International
dc.rights.urihttps://creativecommons.org/licenses/by/4.0/
dc.titleTowards a therapy for mitochondrial disease: an update
dc.typeArticle
prism.endingPage1261
prism.issueIdentifier5
prism.publicationNameBiochemical Society Transactions
prism.startingPage1247-
prism.volume46
dc.identifier.doi10.17863/CAM.33531
dcterms.dateAccepted2018-09-10
rioxxterms.versionofrecord10.1042/BST20180134
rioxxterms.licenseref.urihttp://creativecommons.org/licenses/by/4.0/
rioxxterms.licenseref.startdate2018-09-10
dc.contributor.orcidViscomi, Carlo [0000-0001-6050-0566]
dc.contributor.orcidGarone, Caterina [0000-0003-4928-1037]
dc.identifier.eissn1470-8752
rioxxterms.typeJournal Article/Review
pubs.funder-project-idEuropean Research Council (322424)
pubs.funder-project-idMedical Research Council (MC_UU_00015/8)
pubs.funder-project-idMedical Research Council (MC_EX_MR/P007031/1)
pubs.funder-project-idMRC (MC_UU_00015/8)
cam.issuedOnline2018-10-09


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Attribution 4.0 International
Except where otherwise noted, this item's licence is described as Attribution 4.0 International